SineuGene's Global First-in-Class Gene Therapy SNUG01 for ALS Receives FDA Orphan Drug Designation

On June 25, 2025, SNUG01, the world’s first gene therapy targeting TRIM72 independently developed by SINEUGENE, was officially granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease.

The FDA’s Orphan Drug Designation is intended to encourage the development of innovative therapies for rare diseases (defined in the U.S. as conditions affecting fewer than 200,000 people). With this designation, SNUG01 will be eligible for a 25% tax credit on clinical research expenses, effectively reducing R&D costs; secondly, it will waive up to $3 million in fees for Biologics License Applications (BLA); thirdly, the drug will enjoy seven years of market exclusivity upon approval. These preferential policies will help accelerate the market launch of SNUG01, ultimately benefiting ALS patients worldwide who urgently need effective treatments.

This FDA Orphan Drug Designation marks another significant milestone following the U.S. FDA’s approval of SNUG01 for clinical trials. It not only fully demonstrates the drug’s substantial potential to address unmet clinical needs in ALS, but also provides strong momentum for the upcoming international, multicenter, registrational Phase I/IIa clinical trial and the global development strategy for SNUG01. The project aims to systematically evaluate the safety, tolerability, and preliminary efficacy of SNUG01 in adult patients with ALS, striving to achieve the therapeutic goal of simultaneous benefit for patients in both China and the United States.

SNUG01 is a first-in-class gene therapy product developed by SINEUGENE based on its AAV technology platform. It is the world’s first gene therapy product targeting TRIM72 as the gene of interest (GOI). This target was identified through an ALS model constructed by Professor Ji Yichang’s laboratory at the School of Basic Medical Sciences, Tsinghua University, using next-generation site-specific knock-in technology. SNUG01 utilizes recombinant adeno-associated virus serotype 9 (rAAV9) as a vector to precisely deliver the human TRIM72 gene to neurons via intrathecal (IT) injection. Preclinical studies indicate that TRIM72 may protect neurons and delay motor neuron degeneration in ALS patients through multiple mechanisms, including membrane repair, antioxidant/mitochondrial function restoration, and reduction of stress granule formation. Completed investigator-initiated trials (IITs) of SNUG01 have preliminarily demonstrated favorable safety and tolerability, along with positive signals in efficacy endpoints and biomarker improvements. Unlike therapies targeting only specific genetic mutations associated with ALS, SNUG01 holds the potential to benefit a broader population of ALS patients due to its multi-dimensional neuroprotective mechanisms, particularly offering a potential solution for sporadic ALS patients, who account for 90% of all cases and currently lack effective treatment options.

ALS is a progressive, fatal neurodegenerative disease that affects both upper and lower motor neurons. Patients experience progressively worsening muscle weakness and atrophy, ultimately impairing swallowing and respiratory functions. As one of the most common motor neuron diseases in adults, it has a median survival time of only 3–5 years. Currently, there is no cure worldwide, and existing therapies can only modestly slow disease progression.

SineuGene, established in late 2021, is a biopharmaceutical company focused on gene therapy for neurological disorders. The company’s lead pipeline originates from over a decade of foundational research in neuroscience conducted by Professor Ji Yichang’s laboratory at the School of Medicine, Tsinghua University. SineuGene has established novel knock-in animal disease model platforms using Drosophila, mice, and Bama miniature pigs to faithfully recapitulate disease characteristics and identify more reliable innovative drug targets. Meanwhile, it has developed a central nervous system (CNS) AAV screening platform to create novel AAV serotypes with high targeting specificity, efficient delivery, and low immunogenicity. Additionally, the company has built a CNS small nucleic acid drug R&D platform encompassing target screening, sequence design optimization, nucleic acid synthesis, chemical modification, and delivery technology development. Leveraging technologies such as AAV-mediated gene expression and small nucleic acid-mediated gene regulation, SineuGene has deployed multiple product pipelines dedicated to tackling neurological diseases including amyotrophic lateral sclerosis (ALS), stroke, Parkinson’s disease (PD), Alzheimer’s disease (AD), multiple system atrophy (MSA), spinocerebellar ataxia type 3 (SCA3), Huntington’s disease (HD), and autism spectrum disorder (ASD). Currently, its lead candidate SNUG01, indicated for ALS, has initiated an investigator-initiated trial (IIT) at Peking University Third Hospital and received Investigational New Drug (IND) clearance from the U.S. FDA on March 21, 2025.